[Clinical features and prognosis of juvenile myelomonocytic leukemia: an analysis of 63 cases].

OBJECTIVES

To investigate the clinical features of juvenile myelomonocytic leukemia (JMML) and their association with prognosis.

METHODS

Clinical and prognosis data were collected from the children with JMML who were admitted from January 2008 to December 2016, and the influencing factors for prognosis were analyzed.

RESULTS

A total of 63 children with JMML were included, with a median age of onset of 25 months and a male/female ratio of 3.2∶1. JMML genetic testing was performed for 54 children, and mutation was the most common mutation and was observed in 23 children (43%), among whom 19 had mutation alone and 4 had compound mutation, followed by mutation observed in 14 children (26%), among whom 12 had mutation alone and 2 had compound mutation. The 5-year overall survival (OS) rate was only 22%±10% in these children with JMML. Of the 63 children, 13 (21%) underwent hematopoietic stem cell transplantation (HSCT). The HSCT group had a significantly higher 5-year OS rate than the non-HSCT group (46%±14% vs 29%±7%, <0.05). There was no significant difference in the 5-year OS rate between the children without gene mutation and those with gene mutation (30%±14% vs 27%±10%, >0.05). The Cox proportional-hazards regression model analysis showed that platelet count <40×10/L at diagnosis was an influencing factor for 5-year OS rate in children with JMML (<0.05).

CONCLUSIONS

The gene was the most common mutant gene in JMML. Platelet count at diagnosis is associated with the prognosis in children with JMML. HSCT can improve the prognosis of children with JMML.